Ignite DMD: Data and clinical trial update

Solid Biosciences announced that they have resumed safe dosing at the 2E14 vg/kg dose, and that this patient experienced mild to moderate adverse events all of which have fully resolved. They are encouraged with the improved safety profile of SGT-001 under our previously reported amended protocol and second-generation manufacturing process, both of which were designed to enhance patient safety.

The data reported were collected from the first six patients dosed in IGNITE DMD twelve to twenty-four months after treatment and include data from three patients dosed at the low dose (5E13vg/kg) and three patients dosed at the high dose (2E14 vg/kg). Data from the delayed treatment cohort, analyzed as an untreated control cohort, were evaluated alongside representative natural history data. The six patients ranged in age from five to 14-years-old at time of dosing.

View the full press release here